FARA's Mission and Organization

The Friedreich’s Ataxia Research Alliance (FARA) is a national, public, 501(c)(3), non-profit, tax-exempt organization dedicated to the pursuit of scientific research leading to treatments and a cure for Friedreich’s ataxia.

FARA's Mission is to marshal and focus the resources and relationships needed to cure FA by raising funds for research, promoting public awareness, and aligning scientists, patients, clinicians, government agencies, pharmaceutical companies and other organizations dedicated to curing FA and related diseases.

FARA focuses on grant making for FA research and building collaborations with organizations dedicated to advancing treatments for FA.

Due to the progressive nature of the disease and the promise of treatments in development, there is urgency to our efforts. Directing attention and resources to FA research and partnering with others that share this commitment, FARA believes it can help bring forward effective treatments and a cure for FA.

Grant Program- FARA supports research by funding and facilitating a competitive and highly regarded grant making program that supports greater than 4 million dollars in research annually and focuses on translational and clinical research (moving discoveries through development to clinical improvements for patients).

Clinical Network & Trials- FARA funds the Collaborative Clinical Research Network in Friedreich's Ataxia (CCRN in FA) - an international network of clinical research centers that work together to advance treatments and clinical care for individuals with Friedreich's ataxia.

Scientific Conference Program- FARA promotes collaboration among scientists, advocating for public-private partnerships that support drug discovery, drug development and clinical research and hosting open forums for leading scientists to share their insights, ideas and challenges to advancing treatments for FA.

Patient Registry- The FARA patient registry is the only worldwide registry of Friedreich’s ataxia patients. This registry currently holds the demographic and clinical information on more than 2,000 Friedreich’s ataxia patients from across the United States and internationally. This registry was created to serve the patient, physician, and research communities.

Advocacy- FARA works cooperatively with government entities and the other organizations that support scientific research aimed at treatments for this disorder. FARA also rallies patients, patient families, scientific investigators, healthcare providers, and others to be supporters and advocates for scientific advancements that will lead to treatments and a cure.

Thanks to the committed efforts of many FA scientists, we now understand the cause of FA and specific mechanisms leading to damage in patients, such as gene mutation, decreased frataxin production, iron sulfur cluster formation, and mitochondrial dysfunction. FARA is supporting the development of treatments aimed at each of these different mechanisms of damage. Because it is based on solid basic science discovery, this targeted approach to treatment has great potential. Furthermore, FA researchers believe that treatment will come in the form of a “cocktail” therapy - meaning that therapies aimed at the different mechanisms of damage have the potential to be used in conjunction with one another to treat the disorder. For further information on this research, visit the Research Pipeline page.

Dr. Harold Varmus, former Director or the National Institutes of Health, gave the following testimony before the House Commerce Subcommitte on Health and Environment in 1998:

"The story of Friedreich's ataxia illustrates how many areas of clinical and basic research can come together in unexpected ways. In this rare disorder, research involving neuroscience, genetics, clinical medicine, molecular biology, and even biology of yeast and bacteria are converging. The findings offer insights to basic biology and to many other disorders, and illustrate the importance of understanding the mechanism of disease in order to devise treatments."

Insights into Friedreich's ataxia could prove beneficial to understandings in other diseases such as:

Muscular Dystrophies (ALS, SMA)

Parkinson's Disease

Huntington's Disease

Alzheimer's Disease

Fragile X Syndrome

Mitochondrial diseases (MELAS, MERF, Lebers)

Ataxia-telangiectasia

Cerebellar ataxias

Diabetes

Stroke

FARA was founded in September 1998 by a group of patient families and three of the world’s leading FA scientists — Drs. Rob Wilson, Bronya Keats, and Massimo Pandolfo. It was staffed and managed as an all volunteer organization until late 2005. FARA's Leadership and numerous patient families and friends have raised critical funds over the years to support FA research. Thanks to the brilliant and committed efforts of many FA scientists, we now understand the cause of FA and specific mechanisms leading to damage in FA patients. These understandings allow for more targeted approaches to treatment which are currently being developed for clinical trials.